Rare disease treatment developer Alnylam Pharmaceuticals Inc. has scrapped plans to begin a Phase III study on use of its second-generation RNAi product Amvuttra (vutrisiran) in a second orphan drug indication because having more than one orphan indication could make the drug subject to Medicare price negotiation under the Inflation Reduction Act.
IRA Effect: Alnylam Acting ‘Rationally’ In Halting Second Orphan Indication For Amvuttra – Analysts
Alnylam decision has been seized by opponents of Medicare price negotiation as evidence the nascent program will undermine innovation, particularly for small molecule drugs.

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